Press Release

Myelodysplastic syndromes (MDS) and acute myeloid leukaemia (AML) are the most common malignant diseases of bone marrow in adults. The prognosis of some patient populations is still poor. In addition, survival rate falls further with increasing age.1

For these risk groups of AML and MDS patients, allogeneic haematopoietic stem cell transplantation (HSCT) is the only potentially curative treatment option.2 However, before receiving healthy donor cells, the patients’ diseased bone marrow cells must first be destroyed. Amongst others, this is done by using what is known as conditioning therapy, by radiation and/or by administering chemotherapeutic agents. This therapy is absolutely essential, but is associated with a high burden and occasionally toxic or even life-threatening side effects for the affected patients.3

There is consequently a great need for effective and, at the same time, less toxic conditioning therapies. This was the inspiration for the largest to date, prospective, randomised, multicentre phase III trial4 in predominantly elderly and, in some cases, comorbid AML and MDS patients requiring allogeneic HSCT. The comparative trial tested a treosulfan-based conditioning regimen as an alternative to the established, reduced-intensity busulfan therapy. The trial was prematurely terminated at the end of last year because the primary objective had already been achieved. Initial results based on the data of 476 patients are now available and will be presented at the 59th Annual Meeting of the American Society of Hematology (ASH) in Atlanta. The abstract will be presented by Prof. Dietrich W. Beelen, University of Duisburg/Essen, the trial leader and representative of the 30 European transplantation centres that are actively participating in the trial.